- ATYR Pharma's Phase 3 EFZO-FIT™ study of **efzofitimod** for pulmonary sarcoidosis **did not meet its primary endpoint** of change from baseline in mean daily oral corticosteroid dose at week 48.  **[1339970_2144291_1]** 
- Despite missing the primary endpoint, the study showed that treatment with **5 mg/kg efzofitimod** resulted in **52.6%** of patients achieving complete steroid withdrawal at week 48, compared to **40.2%** on placebo.  **[1339970_2144291_1]** **[1339970_2144291_2]** 
- Patients treated with **5 mg/kg efzofitimod** also experienced a clinical improvement of **10.36 points** in the King's Sarcoidosis Questionnaire (KSQ) Lung Score, versus **6.19 points** for placebo, and the drug was **well tolerated**.  **[1339970_2144291_1]** **[1339970_2144291_3]** **[1339970_2144291_4]** 
- The primary endpoint was missed due to a **higher-than-anticipated placebo response**, with **40.2%** of placebo patients achieving complete steroid withdrawal, largely attributed to the rigorous study protocol.  **[1339970_2144291_2]** **[1339970_2144291_8]** 
- The company plans to **engage with the FDA** to determine the path forward for efzofitimod in pulmonary sarcoidosis, citing the drug's activity across multiple clinically relevant efficacy endpoints and favorable safety profile.  **[1339970_2144291_1]** **[1339970_2144291_4]**

ATYR Pharma Announces Top-Line Results from Phase 3 EFZO-FIT™ Study

- ATYR Pharma, Inc. announced **topline results** from its **Phase 3 EFZO-FIT™ study** of efzofitimod in **268 patients with pulmonary sarcoidosis** on **September 15, 2025**.  **[1339970_0001193125-25-202865_atyr-20250915.htm:1]** 
- The study **did not meet its primary endpoint** of change from baseline in mean daily oral corticosteroid (OCS) dose at week 48, with a reduction to an average of **2.79 mg for 5.0 mg/kg efzofitimod vs 3.52 mg for placebo (p=0.3313)**.  **[1339970_0001193125-25-202865_atyr-20250915.htm:1]** **[1339970_0001193125-25-202865_atyr-ex99_1.htm:2]** 
- Despite not meeting the primary endpoint, the study demonstrated a **clinical improvement in the King’s Sarcoidosis Questionnaire (KSQ)-Lung score** at week 48 for **5.0 mg/kg efzofitimod compared to placebo (p=0.0479)**.  **[1339970_0001193125-25-202865_atyr-20250915.htm:1]** **[1339970_0001193125-25-202865_atyr-ex99_1.htm:2]** 
- Additionally, a responder analysis showed that **29.5% of patients on 5.0 mg/kg efzofitimod achieved complete steroid withdrawal with KSQ-Lung improvement vs 14.4% in placebo (p=0.0199)**.  **[1339970_0001193125-25-202865_atyr-20250915.htm:1]** **[1339970_0001193125-25-202865_atyr-ex99_1.htm:2]** 
- The company plans to **engage with the U.S. Food and Drug Administration (FDA)** to review the results and determine the path forward for efzofitimod in pulmonary sarcoidosis, believing the findings indicate drug activity across multiple clinically relevant efficacy endpoints.  **[1339970_0001193125-25-202865_atyr-20250915.htm:1]** **[1339970_0001193125-25-202865_atyr-ex99_1.htm:2]** **[1339970_0001193125-25-202865_atyr-20250915.htm:2]**

aTYR PHARMA (ATYR)